Towards a Cure – new project

Robin Bligh, HSPRF President – on a new project under consideration. 

We have developed tests for 3 important genes and these will be available in November. It is now time to start switching research priority to finding an HSP cure (no, not utopia) finding a therapeutic drug with the help of stem cell research.

Stem cell science will enable scientists to demonstrate (without experimental animals) the growth of cell lines developed from CNS (central nervous system) stem cells from HSP SPG4 patients on the one hand and from unaffected people on the other. The purpose is to observe the biological differences. 

Your Australian HSP Research Foundation has developed a proposal in conjunction with the National Centre for Adult Stem Cell Research (NCASCR), centred at Griffith University in Queensland, to conduct a Pilot Study aimed at:-

  • Growing and maintaining CNS (olfactory) stem cell lines
  • Differentiating them to other nerve cells
  • Defining biological differences in the HSP SPG4 cell line from the unaffected cell line.

The proposal involves the use of adult stem cells as disease models. Even though adult stem cells are ideally suited for use in cell transplant therapy because there is no patient rejection, this proposal does not encompass cell regeneration.

The study is to last 12 months and cost $100,000. Upon achievement of those goals it means that Griffith University will make an NHMRC submission for a grant to identify and validate a Drug Target.

Then, knowing the Drug Target, the next stage is to screen thousands of compounds from the Griffith Uni compound library using modern high throughput screening methods to find active compounds that could be developed as therapeutic drugs.

Ten years ago we were saying “wish we had a therapeutic drug” and we are still saying that. Is it time to make a start or should we wait another 10 years?

If the long term goal of finding, proving and developing a therapeutic drug is successful then it can benefit HSPers the world over. Consequently your HSP Research Foundation put a collaborative proposal to the HSP group in 9 different countries.  Interest has been expressed by 2 countries and we are now developing a more definitive proposal in conjunction with the NCASCR towards a collaborative project that would share the scientific outcome and the cost.  This is still in progress.